Success in Kadimastem’s First Pre-Clinical Trial for Treatment of Amyotrophic Lateral Sclerosis (ALS)

June 18, 2014

Success in Kadimastem’s First Pre-Clinical Trial for Treatment of Amyotrophic Lateral Sclerosis (ALS)

When brain supporting cells produced by Kadimastem were injected into the central nervous system of a mouse model of Amyotrophic Lateral Sclerosis (ALS), the mice that received the injections survived longer than those that did not, and there was a significant improvement in the motor function of the treated mice

Nes Ziona, Science Park, June 18 2014, Israeli biotechnology company Kadimastem is reporting success in a preclinical trial for the treatment of ALS.

ALS is the most serious of a group of degenerative diseases that damage the motor neurons, which control most of the muscles in the body. The destruction of these neurons causes complete paralysis of the muscles they control. There is no cure or any significant drug treatment today for ALS. In recent years, there has been growing scientific evidence that in patients with the disease, the ability of the central nervous system’s support cells (astrocytes) to maintain a growth environment that supports the motor neurons is impaired. Injecting healthy, functioning support cells into the nervous system of ALS patients could provide systemic support for the damaged motor neurons and thereby slow the progression of the disease, improve patients’ quality of life, and extend their lives.

Kadimastem’s unique technology allows it to produce the support cells that will be used to treat patients as an off-the-shelf product for large patient populations. The company has families of patents and patent applications that protect the technology for producing these cells from stem cells.

The trial examined the effectiveness of injecting Kadimastem’s unique support cells (astrocytes) into the spinal fluid of a mouse model of ALS. This model has tremendous significance in predicting the effectiveness of the treatment in human beings. The results of the trial showed an increased life expectancy for the mice injected with the cells. In addition, there was a significant improvement in the motor (muscle) function of the treated mice compared to those that were untreated. The effectiveness of the treatment is also reflected in other indices indicating a delay in disease onset. Injections into spinal fluid are a standard procedure done routinely in hospitals around the world. Kadimastem found that such injections enable the cells to disperse throughout the central nervous system, and it thus established the method of cell penetration in future treatment of patients.

In light of the positive results of the preclinical trial, Kadimastem intends to conduct preliminary talks (pre-IND) with regulatory authorities such as the U.S. FDA to pave the regulatory path to conducting clinical trials.

Kadimastem CEO Yossi Ben-Yossef noted that “Our company is very satisfied with the results of the preclinical trial, which show that our product has therapeutic effects on the ALS mouse model. We intend to give priority to this project and to conduct a dialogue with the FDA and other regulatory authorities in order to advance it to the clinic as soon as possible. In addition, we will expedite Kadimastem’s contacts with large pharmaceutical companies and other players that will be appropriate strategic partners for advancing the product to the clinic quickly.”

Prof. Michel Revel, Kadimastem’s Chief Scientist, stated that “We are very pleased with the results of the trial, which prove the efficacy of the cell therapy on the ALS mouse model. These encouraging results will enable us to continue to make progress in developing the cell therapy, which is based on the use of brain supporting cells. These can be produced from pluripotent stem cells on an industrial scale using the company’s unique technology.”

Prof. Tamir Ben-Hur, head of the Department of Neurology at Hadassah Ein Karem Hospital and a world expert in neurological diseases, who serves as a member of Kadimastem’s Scientific Advisory Board, added that “The results presented by the company are impressive and unique, in particular when they are compared with results reported until now in other preclinical trials. The data in the trial prove that the cells implanted in the ALS mice had a therapeutic effect that led to the slowing of disease progression. Of course, additional development work is necessary before the clinical stage.”

About Kadimastem

Kadimastem (www.kadimastem.com) (TASE: KDST) develops human stem cells used to create medical solutions for diabetes and degenerative diseases of the nervous system and for drug screening. The company’s technological platform makes it possible to differentiate stem cells into a range of functional human cells, including neuron-supporting cells in the brain as well as pancreatic cells that secrete insulin – beta cells.

Kadimastem relies on technology that was initially developed at the Weizmann Institute in Prof. Michel Revel’s laboratory, and which are currently developed and advanced in the company’s labs at the Weizmann Science Park. Prof. Revel, who currently serves as the company’s Chief Scientist, is the developer of the Rebif®, the blockbuster drug for the treatment of Multiple Sclerosis. Kadimastem is developing two types of medical applications: A. Regenerative medicine, whose purpose is to repair and replace tissues and organs that have been damaged by illness using healthy cells grown in a laboratory conditions, for example, transplantation of insulin-secreting pancreas cells to treat insulin-dependent diabetes, or transplantation of healthy neural cells to support survival of nerve cells and treat ALS; B. A platform for drug screening, which uses functional human cells and tissues to discover new medicines.